Pipeline

Data from Solid’s IGNITE DMD Phase I/II clinical trial evaluating the first-generation candidate SGT-001, which used the AAV9 capsid, showed that expression of the nNOS microdystrophin transgene may have the potential to slow or stop the progression of Duchenne. In studies of SGT-003 performed in the mdx mouse model of Duchenne and in vitro in human Duchenne cell lines, multiple fold increases in microdystrophin expression have been observed in comparison to AAV9, suggesting that therapeutic dose levels using AAV-SLB101 can be lower than first generation candidates.

Microdystrophin is a synthetic form of the dystrophin gene that was designed because the natural, full-size dystrophin gene is too big to fit into an AAV vector. Microdystrophin retains the most critical components of full-size dystrophin yet fits into an AAV vector.

Researchers are currently studying several different microdystrophin genes for their potential to drive production of functional versions of dystrophin protein in muscles of the body.

AVB-202-TT is a novel gene transfer product candidate being developed for the treatment of Friedreich’s ataxia. Friedreich’s ataxia is a rare inherited genetic disease caused by loss of frataxin with both neurological and cardiac manifestations affecting muscle control and coordination, with possible loss of vision and hearing and slurred speech. AVB-202-TT, like other potential gene replacement treatments for Friedreich’s ataxia, is intended to replace the frataxin gene across relevant tissues, with the goal of preventing progression or reversing the course of the disease. AVB-202-TT is in preclinical development and utilizes a dual route of administration (via both intrathecal and intravenous routes) to more rigorously target disease pathology. Preclinical data from three animal models, including mouse and nonhuman primate, supported preclinical proof of concept. Early findings in preclinical studies demonstrated improved survival and cardiac function, as well as mitochondrial function in mice.

SGT-501 is in development for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CASQ2 & RYR2 proteins regulate cardiac calcium (Ca2+), important for electrical conduction and cardiac contraction / relaxation.

SGT-501 is in development for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CASQ2 & RYR2 proteins regulate cardiac calcium (Ca2+), important for electrical conduction and cardiac contraction / relaxation.

AVB-401 is a novel gene transfer product candidate being developed for the treatment of BAG3 mediated dilated cardiomyopathy (BAG3). BAG3 is a rare cardiac disease and is characterized by mutations in the BAG3 gene. Sufficient levels of functional BAG3 are required for healthy cardiac function.